TY - JOUR. T1 - Analysis of the N-glycans of recombinant human Factor IX purified from transgenic pig milk. AU - Gil, Geun Cheol. AU - Velander, William H.. AU - Van Cott, Kevin E.. PY - 2008/7/1. Y1 - 2008/7/1. N2 - Glycosylation of recombinant proteins is of particular importance because it can play significant roles in the clinical properties of the glycoprotein. In this work, the N-glycan structures of recombinant human Factor IX (tg-FIX) produced in the transgenic pig mammary gland were determined. The majority of the N-glycans of transgenic pig-derived Factor IX (tg-FIX) are complex, bi-antennary with one or two terminal N -acetylneuraminic acid (Neu5Ac) moieties. We also found that the N-glycan structures of tg-FIX produced in the porcine mammary epithelial cells differed with respect to N-glycans from glycoproteins produced in other porcine tissues. tg-FIX contains no detectable Neu5Gc, the sialic acid commonly found in porcine glycoproteins produced in other tissues. Additionally, we ...

In previous work we transferred a human factor IX-encoding adeno-associated viral vector (AAV) into skeletal muscle of men with severe hemophilia B. Biopsy of injected muscle up to 1 year after vector injection showed evidence of gene transfer by Southern blot and of protein expression by IHC and immunofluorescent staining. Although the procedure appeared safe, circulating F.IX levels remained subtherapeutic (| 1%). Recently, we obtained muscle tissue from a subject injected 10 years earlier who died of causes unrelated to gene transfer. Using Western blot, IHC, and immunofluorescent staining, we show persistent factor IX expression in injected muscle tissue. F.IX transcripts were detected in injected skeletal muscle using RT-PCR, and isolated whole genomic DNA tested positive for the presence of the transferred AAV vector sequence. This is the longest reported transgene expression to date from a parenterally administered AAV vector, with broad implications for the future of muscle-directed gene

Hypersensitivity or allergic reactions (such as angioedema, burning and tingling at the injection site, chills, redness, generalized urticaria, headache, urticarial reactions, low blood pressure, lethargy, nausea, agitation, tachycardia, chest tightness, tingling), vomiting, asthmatic respiration) have been observed inconsistently in some patients treated with human coagulation factor IX preparations.. In some cases, these reactions have resulted in a severe anaphylactic reaction, associated with the simultaneous appearance of factor IX inhibitor (see Warnings and Precautions Special warnings and special precautions for use section). ). The required treatment depends on the nature and severity of the reaction.. The occurrence of nephrotic syndrome has been described following the induction of immune tolerance in hemophilia B patients with a factor IX inhibitor.. Rare cases of hyperthermia can be observed.. Patients with hemophilia B may develop anti-factor IX antibodies (inhibitors).. The ...

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TY - JOUR. T1 - Physiological levels of blood coagulation factors IX and X control coagulation kinetics in an in vitro model of circulating tissue factor. AU - Tormoen, Garth W.. AU - Khader, Ayesha. AU - Gruber, András. AU - McCarty, Owen J.T.. PY - 2013/6/1. Y1 - 2013/6/1. N2 - Thrombosis significantly contributes to cancer morbidity and mortality. The mechanism behind thrombosis in cancer may be circulating tissue factor (TF), as levels of circulating TF are associated with thrombosis. However, circulating TF antigen level alone has failed to predict thrombosis in patients with cancer. We hypothesize that coagulation factor levels regulate the kinetics of circulating TF-induced thrombosis. Coagulation kinetics were measured as a function of individual coagulation factor levels and TF particle concentration. Clotting times increased when pooled plasma was mixed at or above a ratio of 4:6 with PBS. Clotting times increased when pooled plasma was mixed at or above a ratio of 8:2 with factor ...

ATLANTA--(BUSINESS WIRE)-- Bioverativ Inc. (NASDAQ: BIVV), a global biopharmaceutical company dedicated to transforming the lives of people with rare blood disorders, today announced findings from a novel imaging study investigating extravascular distribution of factor IX therapies, including its leading extended half-life therapy, ALPROLIX® Antihemophilic Factor IX (Recombinant), Fc Fusion Protein. The preclinical study showed ALPROLIX had a higher level of extravascular distribution and retention in certain joint areas when compared with conventional factor IX and a glycoPEGylated factor IX analog. The study was conducted in collaboration with Invicro, LLC, a leading imaging services provider, and is being presented today at the 59th Annual Meeting of the American Society of Hematology. Joint disease, which is caused by frequent bleeding into joints over time, is one of the most common complications for people with hemophilia and often results in chronic pain and significant disability. The ...

GAITHERSBURG, Md., April 30, 2015-- Emergent BioSolutions Inc. today announced that the U.S. Food and Drug Administration has approved IXINITY ®, an intravenous recombinant human coagulation factor IX therapeutic for the control and prevention of bleeding episodes and for perioperative management in adults and children,≥ 12 years of age, with Hemophilia B....

GMAb Factor IX antibody is produced in vitro, under serum-free conditions, and purified by Protein G affinity chromatography. Custom conjugation available.

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... DEERFIELD Ill. Jan. 7 /- Baxter Internati... Extending our recombinant portfolio beyond ADVATE which is indicated...Pre-clinical work on a longer-acting version of recombinant Factor IX...,Baxter,Announces,Recombinant,Factor,IX,Development,Program,For,Hemophilia,B,medicine,advanced medical technology,medical laboratory technology,medical device technology,latest medical technology,Health

We found sustained therapeutic expression of factor IX coagulant activity after gene transfer in 10 participants with hemophilia who received the same vector dose. Transgene-derived factor IX coagulant activity enabled the termination of baseline prophylaxis and the near elimination of bleeding and …

PLAINSBORO, N.J., May 31, 2017 /PRNewswire/ - Novo Nordisk today announced that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application for REBINYN® (Coagulation Factor IX (Recombinant), GlycoPEGylated) for the treatment of adults and children with hemophilia B. Hemophilia B is a chronic and inherited bleeding …. ...

Background. Hemophilia is a rare recessive X-linked disease characterized by a deficiency of coagulation factor VIII or factor IX. Its current treatment is merely palliative. Advanced therapies are likely to become the treatment of choice for the disease as they could provide a curative treatment. Methods. The present study looks into the use of a safe non-viral transfection method based on nucleofection to express and secrete human clotting factor IX (hFIX) where human adipose tissue derived mesenchymal stem cells were used as target cells in vitro studies and NOD. Cg-Prkdcscid Il2rgtm1Wjl/SzJ mice were used to analyze factor IX expression in vivo studies. Previously, acute liver injury was induced by an injected intraperitoneal dose of 500 mg/kg body weight of acetaminophen. Results. Nucleofection showed a percentage of positive cells ranging between 30.7% and 41.9% and a cell viability rate of 29.8%, and cells were shown to secrete amounts of hFIX between 36.8 and 71.9 ng/mL. hFIX levels in the blood

A sequence of about forty amino-acid residues found in epidermal growth factor (EGF) has been shown [ (PUBMED:2288911) (PUBMED:6334307) (PUBMED:3534958) (PUBMED:6607417) (PUBMED:3282918) ] to be present in a large number of membrane-bound and extracellular, mostly animal, proteins. Many of these proteins require calcium for their biological function and a calcium-binding site has been found at the N terminus of some EGF-like domains [ (PUBMED:1527084) ]. Calcium-binding may be crucial for numerous protein-protein interactions. For human coagulation factor IX it has been shown [ (PUBMED:7606779) ] that the calcium-ligands form a pentagonal bipyramid. The first, third and fourth conserved negatively charged or polar residues are side chain ligands. The latter is possibly hydroxylated (see aspartic acid and asparagine hydroxylation site) [ (PUBMED:1527084) ]. A conserved aromatic residue, as well as the second conserved negative residue, are thought to be involved in stabilising the calcium-binding ...

A sequence of about forty amino-acid residues found in epidermal growth factor (EGF) has been shown [ (PUBMED:2288911) (PUBMED:6334307) (PUBMED:3534958) (PUBMED:6607417) (PUBMED:3282918) ] to be present in a large number of membrane-bound and extracellular, mostly animal, proteins. Many of these proteins require calcium for their biological function and a calcium-binding site has been found at the N terminus of some EGF-like domains [ (PUBMED:1527084) ]. Calcium-binding may be crucial for numerous protein-protein interactions. For human coagulation factor IX it has been shown [ (PUBMED:7606779) ] that the calcium-ligands form a pentagonal bipyramid. The first, third and fourth conserved negatively charged or polar residues are side chain ligands. The latter is possibly hydroxylated (see aspartic acid and asparagine hydroxylation site) [ (PUBMED:1527084) ]. A conserved aromatic residue, as well as the second conserved negative residue, are thought to be involved in stabilising the calcium-binding ...

CheckOrphan is a non-profit organization located in Basel, Switzerland and Santa Cruz, California that is dedicated to rare, orphan and neglected diseases. CheckOrphan offers users an interactive and dynamic platform for all these diseases. This strategy allows visitors to be updated daily on all the latest news and interact with people internationally. This is essential, because due to the nature of these diseases, there is not a large concentration of individuals within any given proximity ...

Factor IX Genetically Deficient (Knockout) Products are applicable to a wide range of in vitro research applications (including ELISA and Western Blot) and for many different areas of research, including coagulation and fibrinolysis. Factor IX deficiency is an animal model for Hemophilia B (Christmas Disease). Our Fact

D. A. Ribeiro, D. F. Passos, H. C. Ferraz, L. R. Castilho Journal of Chromatography B, 938 (2013) 111-118 Both recombinant and plasma-derived factor IX

Sheep Anti Human Factor IX Polyclonal Fractionated from Innovative Research is a polyclonal antibody in a Frozen liquid format. This antibody has been purified using protein G affinity chromatography . Fractionated preparations may be especially useful in research where high affinity is necessary, especially in cases w

Learn about ALPROLIX, a therapy regimen for the treatment of hemophilia B that uses Fc Fusion to keep Factor IX circulating in your bloodstream longer

Background.Recently a novel bifunctional antibody (emicizumab) that binds both factor IXa (FIXa) and factor X (FX) has been used to treat hemophilia A. Emicizum

Anti-Mouse Factor IX, Clone 1 - Detects Mouse Factor IX and Factor IXa in ELISA, IX and IXa heavy chain reduced and non-reduced in western blot. Host rat.

The N-terminal EGF domain has been shown to at least in part be responsible for binding tissue factor.[6] Wilkinson et al. conclude that residues 88 to 109 of the second EGF domain mediate binding to platelets and assembly of the factor X activating complex.[7] The structures of all four domains have been solved. A structure of the two EGF domains and the trypsin-like domain was determined for the pig protein.[8] The structure of the Gla domain, which is responsible for Ca(II)-dependent phospholipid binding, was also determined by NMR.[9] Several structures of super active mutants have been solved,[10] which reveal the nature of factor IX activation by other proteins in the clotting cascade. ...

Anti-Mouse Factor IX, Clone 2 - Detects Mouse Factor IX and Factor IXa in ELISA and non-reduced in western blot, does not cross react with human. Host rat.

Carcao M, Moorehead P, Lillicrap D. Hemophilia A and B. In: Hoffman R, Benz EJ Jr, Silberstein LE, Heslop HE, Weitz JI, Anastasi J, eds. Hematology: Basic Principles and Practice. 6th ed. Philadelphia, PA: Elsevier Saunders; 2013:chap 137.. Chernecky CC, Berger BJ. Factor IX (Christmas factor, hemophilic factor B, plasma thromboplastin component, PTC) - blood. In: Chernecky CC, Berger BJ, eds. Laboratory Tests and Diagnostic Procedures. 6th ed. St Louis, MO: Elsevier Saunders; 2013:505-506.. Schmaier AH. Laboratory evaluation of hemostatic and thrombotic disorders. In: Hoffman R, Benz EJ Jr, Silberstein LE, Heslop HE, Weitz JI, Anastasi J, eds. Hematology: Basic Principles and Practice. 6th ed. Philadelphia, PA: Elsevier Saunders; 2013:chap 131. ...

The current study used adeno-associated virus (AAV) 8 to deliver the Factor IX gene along with additional genetic material into the patients liver. AAV8 was picked because the incidence of natural infection with AAV8 is low. It belongs to a family of viruses that target liver cells, but do not cause disease in humans or integrate into human DNA. Participants in this study received no immune suppressing drugs prior to gene therapy. This approach was jointly pioneered by St. Jude and UCL, initially in the laboratory of study co-author Professor Arthur Nienhuis a member of the St. Jude Department of Hematology. For this study, each patient received a one-time infusion of the vector into a vein in the arm. Two patients each were treated with escalating doses of the vector. Following treatment, Factor IX levels rose in all six patients from less than 1% of normal levels prior to the gene therapy to between 2 and 12%.. Factor IX levels increased the most in the two study volunteers who received the ...

Prophylactic regimens resulted in low single-digit Median dosing interval was 14 days in the individualized interval prophylaxis arm during the last 6 months on

Scientists working in Oxford have made major contributions to this field. Robert Gwynne Macfarlane was instrumental in identifying the clotting cascade. George Brownlee first cloned the Factor IX gene, leading to the production of recombinant Factor IX now used to treat patients with haemophilia B. Sir David Weatherall pioneered research into the inherited basis of blood disorders, particularly thalassaemia, culminating in a prestigious Lasker Award in 2010. Current research in haematology is concentrated in the MRC Molecular Haematology Unit housed in the Weatherall Institute of Molecular Medicine and the Nuffield Department of Clinical Laboratory Sciences. Areas of strength include the regulation of gene expression in haematopoiesis, understanding haematopoietic stem cells and how they are perturbed in various form of leukemia. ...

Inclusion Criteria: - Male patients with age ≥ 20 years old - Endogenous FIX activity ≤1 IU/dL, - At least 50 exposure days (EDs) with FIX products, - No detectable inhibitor to FIX or inhibitor history, - Had a minimum of 2 nontrauma-induced bleeding episodes (any type or location) treated in the 6 months preceding study entry, - The patient or patients parents or legal authorized representative, as applicable, are capable to understand the study objectives and procedure, and sign the written consent, - Accept that the supply of Idelvion might be stopped once the study is completed, - Able to complete a diary during 12 months or 50 EDs, whichever comes first. Exclusion Criteria: - Currently participating in an interventional clinical trial, - Known hypersensitivity to any FIX product or hamster protein, - Known inhibitor to FIX or inhibitor history, - With other comorbidities which are not suitable for this study, at investigators discretion, - Not able to compliant with the prophylactic ...

Post-transfusion hepatitis is a serious and frequent complication in patients treated with clotting factor concentrates made from plasma pooled from a large number of donors. The occurrence is close to 100% in hemophiliacs who have not previously received blood or blood derivatives (first-exposure patients) (1-3). Recently, several manufacturers of concentrate have developed physical or chemical methods that remove or inactivate viruses with little or no loss of clotting factor activity. One manufacturer has added hydrophobic interaction chromatography on octanohydrazide-agarose to the regular fractionation process of a commercial concentrate of clotting factors II, VII, IX, and X (4). In-vitro studies have ...

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The aim of this study was to characterize the variability of bleeding phenotype and its association with plasma factor IX coagulant activity (FIX:C) in haemophilia B carriers in a large Amish pedigree with a unifying genetic mutation, C-to-T transition at base 31008 of the factor IX gene (Xq27.1-27.2). A cross-sectional ...

Ultrasound-guided percutaneous delivery of adenoviral vectors encoding the beta-galactosidase and human factor IX genes to early gestation fetal sheep in utero.

Gentaur molecular products has all kinds of products like :search , Molecular Innovations \ Anti Mouse Factor IX and IXa \ MA-MFIX-2 for more molecular products just contact us

Easy-to-read patient leaflet for Factor IX (Recombinant [Fc Fusion Protein]). Includes indications, proper use, special instructions, precautions, and possible side effects.

Find everything you need to know about Factor IX Complex, including what it is used for, warnings, reviews, side effects, and interactions. Learn more about Factor IX Complex at EverydayHealth.com.

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FITC Labeled Anti mouse factor IX von Molecular Innovations bei SZABO-SCANDIC erhältlich. Weiteres zu Primärantikörper finden Sie hier.

December 8, 2015. Note: The following is the edited version of a press release issued by CSL Behring. The original release can be found here. On December 7, 2015, CSL Behring presented data from its Phase III PROLONG-9FP clinical program evaluating the efficacy and long-term safety of its investigational long-acting fusion protein linking recombinant coagulation factor IX with recombinant […]. ...

Looking for online definition of coagulation factor IX in the Medical Dictionary? coagulation factor IX explanation free. What is coagulation factor IX? Meaning of coagulation factor IX medical term. What does coagulation factor IX mean?

article{1e9e7fa6-4990-4ef4-af96-b18ff9968d5d, abstract = {,p,Anaphylaxis/anaphylactoid reactions have recently been reported after few treatments with factor IX concentrates in patients with haemophilia B at the same time as inhibitors to factor IX were demonstrated. In some of these cases nephrotic syndrome has appeared during immune tolerance induction (ITI) with high doses of factor IX concentrates. Gene deletions seem to be associated with a high risk of developing antibodies to factor IX. This report presents two brothers with deletion of 1 bp in exon f of the factor IX gene. Both showed anaphylactoid reactions and they were desensitized using slow i.v. injections of factor IX. At the time of anaphylaxis, inhibitors of factor IX in a low titre could be demonstrated. The elder brother responded well after a short time on ITI and has no spontaneous bleedings on regular prophylaxis although in a somewhat higher dose than expected. On the other hand, in spite of comparable regimens, the younger ...

TY - JOUR. T1 - Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B. AU - Powell, Jerry S. AU - Pasi, K. John. AU - Ragni, Margaret V.. AU - Ozelo, Margareth C.. AU - Valentino, Leonard A.. AU - Mahlangu, Johnny N.. AU - Josephson, Neil C.. AU - Perry, David. AU - Manco-Johnson, Marilyn J.. AU - Apte, Shashikant. AU - Baker, Ross I.. AU - Chan, Godfrey C.. AU - Novitzky, Nicolas. AU - Wong, Raymond S.. AU - Krassova, Snejana. AU - Allen, Geoffrey. AU - Jiang, Haiyan. AU - Innes, Alison. AU - Li, Shuanglian. AU - Cristiano, Lynda M.. AU - Goyal, Jaya. AU - Sommer, Jurg M.. AU - Dumont, Jennifer A.. AU - Nugent, Karen. AU - Vigliani, Gloria. AU - Brennan, Aoife. AU - Luk, Alvin. AU - Pierce, Glenn F.. PY - 2013. Y1 - 2013. N2 - BACKGROUND: Prophylactic factor replacement in patients with hemophilia B improves outcomes but requires frequent injections. A recombinant factor IX Fc fusion protein (rFIXFc) with a prolonged half-life was developed to reduce the frequency of ...

Investigators report no evidence of toxicity in the four hemophilia B patients enrolled to date in a gene therapy trial using a vector under development at St. Jude Childrens Research Hospital and UCL (University College London) to correct the inherited bleeding disorder.. This trial was designed primarily as a safety test, with low and intermediate doses of the vector expected to produce little detectable Factor IX. The Factor IX protein helps the blood form clots. Individual with hemophilia B lack adequate levels of this clotting factor. The first participant in the open-label Phase I/II trial had an unexpectedly high level of Factor IX expression after receiving the lowest dose of the vector being tested in this study. Levels of the Factor IX protein rose from less than 1 percent to 2 percent of normal after the experimental vector was infused into the patient.. The patients Factor IX production remains elevated more than nine months later. Since the infusion the patient has also not ...

Valder Arruda, M.D., Ph.D.. Our laboratory is interested in the development of gene-based strategies for the treatment of bleeding and thrombotic diseases. In a collaborative effort, we, along with others, have carried out early-phase clinical studies on adeno-associated viral (AAV) vectors for the treatment of severe hemophilia B (factor IX deficiency). Current projects are focused on translational research studies on the efficacy and safety of intravascular delivery of AAV vectors to skeletal muscle or liver of dogs and mice with severe hemophilia B and hemophilia A (factor VIII deficiency). We are developing novel variants of coagulation factor VIII or factor IX with enhanced biological activity to optimize gene and protein based strategies. We have identified a factor IX variant (FIX Padua) with 8-10-fold higher specific activity, and this molecule is now used for gene therapy in hemophilia B dog models.. Recently the focus of the laboratory has been on the use of gene therapy to treat a ...

Control and Prevention of Bleeding Episodes in Hemophilia B. BeneFIX®, Coagulation Factor IX (Recombinant), is indicated for the control and prevention of bleeding episodes in adult and pediatric patients with hemophilia B (congenital factor IX deficiency or Christmas disease).. Peri-operative Management in Patients with Hemophilia B. BeneFIX®, Coagulation Factor IX (Recombinant), is indicated for peri-operative management in adult and pediatric patients with hemophilia B.. BeneFIX®, Coagulation Factor IX (Recombinant), is NOT indicated for:. a. treatment of other factor deficiencies (e.g., factors II, VII, VIII, and X),. b. treatment of hemophilia A patients with inhibitors to factor VIII,. c. reversal of coumarin-induced anticoagulation,. d. treatment of bleeding due to low levels of liver-dependent coagulation factors.. For indications, dosing and other information, please refer to the prescribing information.. ...

This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile.. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The pharmaceutical form of AMT-061 is a solution for intravenous infusion administered at a dose of 2 x 10^13 gc/kg. ...

article{6540f780-e849-4367-a13e-127a460cab4a, abstract = {Haemophilia B, an X-linked recessive bleeding disorder characterized by lack or deficiency of factor IX, has been shown to be caused by any of a variety of DNA abnormalities (partial or total deletions, nonsense or missense mutations). Since in most countries carrier detection is based on factor IX coagulant activity (FIX:C) assay, this study was designed to determine whether carriers FIX:C values are dependent on the severity of haemophilia (mild, moderate or severe) or on the genetic anomaly in the family. FIX:C concentrations were studied in 28 obligate carriers, 39 women known to carry the mutation and 33 verified noncarriers subgrouped by severity of disorder or genetic anomaly. No significant subgroup differences in FIX:C values were found, thus suggesting the level of FIX:C concentrations in carriers to be unaffected by the severity of haemophilia, or by its expression (i.e. deficient or dysfunctional factor IX). The specificity ...

TY - JOUR. T1 - Low-factor consumption for major surgery in haemophilia B with long-acting recombinant glycoPEGylated factor IX. AU - Escobar, M. A.. AU - Tehranchi, R.. AU - Karim, F. A.. AU - Caliskan, U.. AU - Chowdary, P.. AU - Colberg, T.. AU - Giangrande, P.. AU - Giermasz, Adam. AU - Mancuso, M. E.. AU - Serban, M.. AU - Tsay, W.. AU - Mahlangu, J. N.. PY - 2017/1/1. Y1 - 2017/1/1. N2 - Introduction: Surgery in patients with haemophilia B carries a high risk of excessive bleeding and requires adequate haemostatic control until wound healing. Nonacog beta pegol, a long-acting recombinant glycoPEGylated factor IX (FIX), was used in the perioperative management of patients undergoing major surgery. Aim: To evaluate the efficacy and safety of nonacog beta pegol in patients with haemophilia B who undergo major surgery. Methods: This was an open-label, multicentre, non-controlled surgery trial aimed at assessing peri- and postoperative efficacy and safety of nonacog beta pegol in 13 previously ...

Hemophilia B is a genetic X-linked bleeding disorder caused by a deficiency in blood-clotting Factor IX (FIX) activity. FIX is synthesized in the liver and circulates in the blood as a proenzyme. Current treatment for hemophilia B is based on replacement of the deficient FIX with IV injections of recombinant FIX protein prophylactically or as needed to treat bleeding episodes. This clinical program will test a gene transfer approach involving the use of a gene delivery vector carrying a FIX gene. This first-in-humans study is intended to evaluate the safety, kinetics, and if possible, the dose of AskBio009 required to achieve stable plasma FIX activity between 10% and 40% of normal activity ...

Hemophilia is a rare genetic bleeding disorder that causes the blood to take a long time to clot as a result of a deficiency in one of several blood clotting factors, and occurs almost exclusively in males. People with hemophilia face specific risks as they are not able to form blood clots efficiently and are at risk for excessive and recurrent bleeding from modest injuries, which have the potential to be life threatening. People with severe hemophilia often bleed spontaneously into their muscles or joints. The incidence of hemophilia B is one in 25,000 male births. People with hemophilia B have a deficiency in clotting factor IX, a specific protein in the blood. Hemophilia B is also called congenital factor IX deficiency or Christmas disease. Current standard of care requires recurrent intravenous infusions of either plasma-derived or recombinant factor IX to control and prevent bleeding episodes. There exists a significant need for novel therapeutics to treat people living with ...

Factor IX Complex is a sterile, lyophilized concentrate composed of a number of Vitamin K-dependent clotting factors found in functioning human plasma. Also known as prothrombin complex concentrate, products containing this complex often include Factor IX (antihemophilic factor B), Factor II (prothrombin), Factor X (Stuart-Prower Factor), and low levels of Factor VII (proconvertin) derived from human plasma. Many commercially available products also contain low levels of other antithrombotic proteins. For example, Kcentra (FDA) also contains the antithrombotic proteins C and S, while Bebulin VH (FDA) contains heparin. Coagulation factors are purified from pooled human plasma and subsequently sterilized and treated. Although Factor IX Complex products contain many different coagulation components, Factor IX is the lead component for potency and efficacy, particularly when used for the treatment of bleeding associated with Hemophilia B (Factor IX deficiency). As the product Kcentra, Factor IX ...

Molecular Analysis of Factor VIII and Factor IX Genes in Hemophilia Patients: Identification of Novel Mutations and Molecular Dynamics Studies

Biogen Idec announced the company submitted a Biologics License Application (BLA) to the FDA for the marketing approval of recombinant factor IX Fc fusion protein (rFIXFc) for the treatment of hemophilia B.

Introduction Despite the advent of extended half-life factor IX (FIX) products with the potential for 14 days or longer prophylactic dosing in patients with severe hemophilia B, long-term data on the use of ≥14-day dosing are lacking for prolonged periods of observation. A post hoc analysis of the B-LONG Phase 3 trial showed that ~50% of subjects in the individualized interval prophylaxis treatment arm (Arm 2, n=29) used extended dosing intervals (≥14 days) and had low annualized bleed rates (ABRs) that were similar to ABRs in subjects on other dosing intervals in Arm 2.1. The purpose of this analysis was to evaluate whether sustained ≥14-day dosing with recombinant FIX Fc fusion protein (rFIXFc) can provide safe and effective protection from bleeding in selected patients with severe hemophilia B over time in a near real-world setting. Methods B-LONG (NCT01027364) enrolled 123 adults and adolescents ≥12 years of age into 1 of 4 treatment arms: weekly prophylaxis (50 IU/kg every 7 days, ...

High-level expression of recombinant human blood coagulation factor in milk of farm animals at a large scale provides a powerful tool for protein production. However, a bottleneck in recent protein synthesis technologies is the high cost of current transgenic livestock system. Here, we report a simple, rapid, and low-cost protein production method based on a replication-defective adenoviral vector system. The recombinant hfVIII adenoviral vector was generated by using homologous recombination in bacteria and transfected the plasmids into the HEK293 packaging cell line. Goat’s mammary glands at the different physiological stage were infected with the recombinant adenovirus containing a human blood coagulation factor VIII gene. The expression level of bioactive hfVIII from milk sample was confirmed by Western blot analysis and ELISA methods. The hfVIII gene was expressed as a protein of about 26 kDa and no recombinant hfVIII protein was detected in negative control treatments. The hfVIII was

An open-label, uncontrolled, single-dose, dose-escalation, multi-centre trial investigating the safety and efficacy of systemic administration of AAV5-hFIX, an adeno-associated viral vector containing a codon-optimised human factor IX gene, to severe haemophilia B ...

Background: Data from earlier hemophilia B (HB) AAV-mediated liver gene transfer trials demonstrated a dose-dependent, capsid-specific immune response that may result in clearance of transduced hepatocytes and loss of transgene expression (Manno et al. 2006, Mingozzi et al. 2007). This has not posed major safety concerns, but may limit efficacy. Prior work incorporated the use of steroids to abort this immune response and maintain factor IX (FIX) expression (Nathwani et al 2014). The percent of transgene expression lost increased with number of elapsed days from transaminase elevation to steroid initiation. Once vector responsiveness to steroids is established, the need for steroids is not a limitation in and of itself but highlights the requirement to reliably and expeditiously recognize an immune response and initiate steroids. Here we present our immunomonitoring data following infusion of SPK-9001, an AAV vector designed to achieve therapeutic FIX:C at a low vector dose to minimize ...

Home Health Care, which includes physical, occupational and speech therapies, as well as nursing, encompasses a broad spectrum of health and social services for recovering, disabled or chronically ill persons... ...

Gly193 substitutions had a modestly larger detrimental effect (1.2-1.5 fold) on cleavage of fIX after Arg180 compared to Arg145 that was associated with varying degrees of fIXα accumulation. Similar effects were noted with substitutions for the adjacent residue Lys192. FXIa with Pro192 cleaved fIX after Arg180 ,10-fold more slowly than after Arg145, generating fIXα with little subsequent conversion to fIXaβ. Cumulatively, these data support the premise that the rates for the two sequential reactions required for normal fIX activation by fXIa are comparable. Therefore, perturbations causing a greater effect on cleavage after Arg180 compared to Arg145, even if relatively small, result in fIXα accumulation. Initial recognition of fIX by fXIa involves substrate binding exosites distinct from the enzyme active site. At least one exosite appears to be located in the fXIa third apple (A3) domain, and may interact with an epitope on the fIX Ca2+-binding Gla-domain. The rate of fIX activation to ...

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August 17, 2015. Note: This is an edited form of a press release from Biogen. To read the original release in its entirety, click here. New clinical data support the long-term safety and efficacy of ALPROLIX® [Coagulation Factor IX (Recombinant), Fc Fusion Protein] in people with severe hemophilia B treated for up to two years, Biogen announced on […]. ...

https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Hemophilia-B Haemophilia B] is a disease caused by a deficiency in the secretion of coagulation factor IX (FIX) which results in the impaired blood coagulation cascade,ref name=ena,Nathwani et al. (2011). Adenovirusassociated virus vector-mediated gene transfer in hemophilia B. N. Engl. J. Med. 365, 2357-2365,/ref,. Thus, the disease is manifested by blood dotting defects. Considering the plasma cells ability to produce de novo protein, it opens up an opportunity for the production of deficient protein and thus the potential cure for protein-deficiency diseases. This review article will focus on the methods used to investigate the ability of activated B cells differentiated from primary naive human B cells to produce functioning factor IX by [https://www.addgene.org/crispr/guide/CRISPR/Cas9 CRISPR/Cas9] genome editing tool and [https://blog.addgene.org/crispr-101-homology-directed-repair homology-directed ...

https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Hemophilia-B Haemophilia B] is a disease caused by a deficiency in the secretion of coagulation factor IX (FIX) which results in the impaired blood coagulation cascade,ref name=ena,Nathwani et al. (2011). Adenovirusassociated virus vector-mediated gene transfer in hemophilia B. N. Engl. J. Med. 365, 2357-2365,/ref,. Thus, the disease is manifested by blood dotting defects. Considering the plasma cells ability to produce de novo protein, it opens up an opportunity for the production of deficient protein and thus the potential cure for protein-deficiency diseases. This review article will focus on the methods used to investigate the ability of activated B cells differentiated from primary naive human B cells to produce functioning factor IX by [https://www.addgene.org/crispr/guide/CRISPR/Cas9 CRISPR/Cas9] genome editing tool and [https://blog.addgene.org/crispr-101-homology-directed-repair homology-directed ...

Regulatory subunit of the blood coagulation factor X- and IX-activating enzyme. The enzyme activates coagulation factor X (F10) by cleaving the Arg-Ile bond and is also able to activate coagulation factor IX (F9) and protein S (PROS1) by specific cleavage of Arg-Ile and Arg-Val bonds. May serve as an exosite by which the enzyme recognizes and binds to the Gla domain of factor X (F10) and factor IX (F9) in a calcium-dependent manner.

Merck & Co. Inc. (NYSE:MRK) said that, it is pressing ahead with Phase III trials aimed at mimicking the worlds biggest selling drug as it looks to create future growth with biosimilars.. Merck & Co. Inc. (NYSE:MRK) belongs to Healthcare sector. Its net profit margin is 11.20% and weekly performance is 2.16%. On last trading day company shares ended up at $52.10. Merck & Co. Inc. (NYSE:MRK) distance from 50-day simple moving average (SMA50) is 2.10%. Biogen Inc. (NASDAQ:BIIB) received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending that marketing authorisation be granted for Alprolix® (rFIXFc), a recombinant factor IX Fc fusion protein therapy for the treatment of haemophilia B. If approved, Alprolix would be among the first therapies in the European Union (EU) to offer people living with haemophilia B prolonged protection against bleeding episodes with prophylactic dosing intervals.. Biogen Inc. ...

Citrated plasma samples should be collected by double centrifugation. Blood should be collected in a blue-top tube containing 3.2% buffered sodium citrate.1 Evacuated collection tubes must be filled to completion to ensure a proper blood to anticoagulant ratio.2,3 The sample should be mixed immediately by gentle inversion at least six times to ensure adequate mixing of the anticoagulant with the blood. A discard tube is not required prior to collection of coagulation samples, except when using a winged blood collection device (ie, butterfly), in which case a discard tube should be used.4,5 When noncitrate tubes are collected for other tests, collect sterile and nonadditive (red-top) tubes prior to citrate (blue-top) tubes. Any tube containing an alternate anticoagulant should be collected after the blue-top tube. Gel-barrier tubes and serum tubes with clot initiators should also be collected after the citrate tubes. Centrifuge for 10 minutes and carefully remove 2/3 of the plasma using a ...

Rabbit recombinant monoclonal Carbonic Anhydrase IX antibody [SP106] validated for IHC, Flow Cyt and tested in Human. Immunogen corresponding to synthetic…

Factor IX - Christmas factor, Plasma thromboplastin component (PTC); intrinsic pathway; deficiency leads to hemophilia B. Factor XI - Plasma thromboplastin antecedent (PTA); intrinsic pathway…

Patient Presentation An 4-year-old male came to clinic in December for his well child care. He was known to have Factor IX deficiency after he had spontaneous bleeding into an elbow as a toddler. He was receiving prophylactic factor treatment at the regional childrens hospital. The past medical history showed no other major bleeding episodes.…

Buy our Recombinant Human Factor XI protein. Ab158409 is a protein fragment produced in Wheat germ and has been validated in WB, ELISA. Abcam provides free…

Used in the treatment of bleeding episodes in hemophilia A or B. NovoSeven is recombinant Factor VIIa and, when complexed with tissue factor can activate coagulation Factor X to Factor Xa, as well as coagulation Factor IX to Factor IXa. Factor Xa, in complex with other factors, then converts prothrombin to thrombin, which leads to the formation of a hemostatic plug by converting fibrinogen to fibrin and thereby inducing local clotting ...

However, clinical superiority is only necessary and applicable if rIX-FP is indeed the same drug as the already approved recombinant factor IX. That is, according to CFR 316.20(b)(5), where the sponsor of a drug that is otherwise the same drug as an already-approved orphan drug seeks orphan designation for the subsequent drug for the same rare disease or condition, an explanation of why the proposed variation may be clinically superior to the first drug should be included in the request for designation. FDA regulations also state that Two protein drugs would be considered the same if the only differences in structure between them were due to posttranslational events or infidelity of translation or transcription or were minor differences in amino acid sequence; other potentially important differences, such as different glycosylation patterns or different tertiary structures, would not cause the drugs to be considered different unless the differences were shown to be clinically superior. It ...

Global biotherapeutics leader CSL Behring today presented data from its Phase III PROLONG-9FP clinical program evaluating the efficacy and long-term safety of its investigational long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP).

Human F10 (Coagulation Factor X) ELISA Kit (EH1065) allows for the in vitro quantitative determination of F10 concentrations in plasma, tissue homogenates and other biological fluids. Manufactured by FineTest.

1. Morfini M. Innovative approach for improved rFVIII concentrate. Eur J Haematol. 2014 Apr 26. 2. Morfini M, Batorova A, Mariani G, Auerswald G, Bernardi F, Di Minno G, Dolce A, Fede C, Giansily-Blaizot M, Ingerslev J, Martinowitz U, Napolitano M, Pinotti M, Schved JF; for the International FVII [IF7] and Seven Treatment Evaluation Registry [STER] Study Groups. Pharmacokinetic properties of recombinant FVIIa in inherited FVII deficiency account for a large volume of distribution at steady state and a prolonged pharmacodynamic effect. Thromb Haemost. 2014 Apr 24;112(2). 3. Hassan HJ, Morfini M, Taruscio D, Abbonizio F, Giampaolo A, Kodra Y, Oliovecchio E, Vittozzi L. Current status of Italian Registries on inherited bleeding disorders. Blood Transfus. 2014 Apr;12 Suppl 3:s576-81. 4. Morfini M, Coppola A, Franchini M, Di Minno G. Clinical use of factor VIII and factor IX concentrates Blood Transfus. 2013 Sep;11 Suppl 4:s55-63.. 5. Santagostino E, Negrier C, Klamroth R, Tiede A, Pabinger-Fasching ...

Haemophilia B is one of the most important inherited disorders of haemostasis in Rhodesian Ridgeback Dogs. The underlying pathomechanism of haemophilia B is a lack or decreased activity of factor IX that plays a critical role in the coagulation cascade. Affected dogs present with hemorrhage that can vary from mild to severe depending on the degree of the disease. The clinical signs include haematomas of large sizes, bleeding of the nose, skin, muscles and joints. If the disease is severe and no precautions are taken, affected dogs can bleed to death after surgery or injuries. ...

1CFH: Structure of the metal-free gamma-carboxyglutamic acid-rich membrane binding region of factor IX by two-dimensional NMR spectroscopy.

Abnova Human F12 Full-length ORF (AAH12390, 1 a.a. - 300 a.a.) Recombinant Protein with GST-tag at N-terminal 25µg Life Sciences:Protein Biology:Proteins:Proteins A-Z:Proteins

PROTHROMBINEX-VF is a freeze-dried preparation of proteins called human prothrombin complex. PROTHROMBINEX-VF contains concentrated factor IX, factor II, factor X and low level of factor VII.

Dommelen, R. van Gedragsveranderingen bij kinderen met ALL behandeld volgens SNWLK ALL 9: een prospectieve longitudinale studie naar Koorts in neutropenische kinderen ten gevolge van chemotherapie; een mogelijke rol voor interleukine-8 Farmacokinetiek en -dynamiek van vincristine bij ALL: een upfront Sedatie en pijnstilling van kinderen met acute lymfatische leukemie (ALL) tijdens beenmergpuncties. Een vergelijkend onderzoek Interactie tussen leukemische cellen en immuun competente cellen Onderzoek naar veiligheid en effectiviteit van recombinant menselijk factor IX bij niet eerder behandelde patiënten met hemofilie B Vroege chronische ITP bij kinderen: intemationale Hoe gaan ouders om met kanker van hun kind; Een vervolgstudie 5 jaar na diagnose Effecten van chronisch gebruik van Monoclate bij kinderen met hemophilie A, die niet eerder zijn behandeld met factor VIII concentraat of andere Een inventarisatie van tumorbiologische kenmerken van hersentumoren op de kinderleeftijd in relatie tot de ...

factor IX sulfation with the TPST-1 constructs, it appears that the C -terminal end of the catalytic domain of TPST could be playing some inhibitory role. We would need

Human F12 partial ORF ( AAH12390, 191 a.a. - 300 a.a.) recombinant protein with GST-tag at N-terminal. (H00002161-Q01) - Products - Abnova

The M.S. in Human Factors covers human-centered concerns - psychological and physiological - during the design and development of systems, products, and work environments.

In the Human Factor, we profile survivors who have overcome the odds. Confronting a life obstacle - injury, illness or other hardship -- they tapped their inner strength and found resilience they didnt know they possessed. This week Chief Medical Correspondent Dr.